BANNOCKBURN, Ill.--(BUSINESS WIRE)--
Baxalta Incorporated (NYSE:BXLT), a global biopharmaceutical leader
dedicated to delivering transformative therapies to patients with orphan
diseases and underserved conditions, today announced that it has
submitted a Marketing Authorization Application (MAA)to the
European Medicines Agency (EMA) for approval of ADYNOVI, an extended
circulating half-life recombinant Factor VIII (rFVIII) treatment,for
pediatric, adolescent and adult patients with hemophilia A and for use
during surgery. Currently licensed in the U.S. as ADYNOVATE
[Antihemophilic Factor (Recombinant), PEGylated] and under regulatory
review in Japan, Canada and Switzerland, ADYNOVI is the only rFVIII
treatment for hemophilia A developed based on the demonstrated efficacy
of ADVATE [Antihemophilic Factor (Recombinant)].

''We are dedicated to bringing ADYNOVI to hemophilia A patients all over
the world,'' said Brian Goff, executive vice president and president,
Hematology, Baxalta. ''As we build on our market-leading portfolio of
direct factor replacement treatments, we continually bring new options
to hemophilia patients, empowering them to manage their hemophilia, with
their caregivers, in the way that works best for them.''

ADYNOVI was studied in patients, 12 to 65 years of age, in a
prospective, global, multi-center, open label, non-randomized study; in
patients 12 years of age and younger, in a prospective, uncontrolled,
open-label, multi-center study; and for the perioperative control of
hemostasis among 15 patients with severe hemophilia A undergoing
surgical procedures. The EMA filing is based on data from these three
Phase 3 clinical trials, which Baxalta shared publicly in December 2015.

Hemophilia A is a challenging chronic disease; treatment regimens
require regular infusions to reduce the risk of bleeding. Working
closely with their health professionals, many patients continue to seek
treatment options that can be better personalized to fit their needs,
providing both effective bleed protection and simplified dosing
schedules. Today, hemophilia affects more than 400,000 people globally
and an estimated 75 percent of people with hemophilia are inadequately
treated.1 Baxalta continues to innovate in order to address
some of the greatest challenges associated with hematologic disorders,
including hemophilia.

About ADYNOVATE in the United States

ADYNOVATE is built on the full-length ADVATE molecule, a leading
treatment for hemophilia A that been used by patients worldwide for more
than 12 years. Through a collaboration with Nektar Therapeutics (NASDAQ:
NKTR), ADYNOVATE leverages proprietary PEGylation technology designed to
extend the amount of FVIII available for use in the body. The technology
was selected because it maintains the integrity of the parent molecule
(ADVATE) and reduces the time at which the body clears ADYNOVATE,
resulting in an extended circulating half-life. This proprietary
technology has been used for more than 15 years in a number of approved
medicines that treat chronic or serious conditions.

Indications:

In the United States, ADYNOVATE, [Antihemophilic Factor (Recombinant),
PEGylated], is a human antihemophilic factor indicated in adolescent and
adult patients (12 years and older) with hemophilia A (congenital factor
VIII deficiency) for:

On-demand treatment and control of bleeding episodes

Routine prophylaxis to reduce the frequency of bleeding episodes

ADYNOVATE is not indicated for the treatment of von Willebrand disease.

Detailed Important Risk Information

CONTRAINDICATIONS

ADYNOVATE is contraindicated in patients who have had prior anaphylactic
reaction to ADYNOVATE, to the parent molecule (ADVATE), mouse or hamster
protein, or excipients of ADYNOVATE (e.g. Tris, mannitol, trehalose,
glutathione, and/or polysorbate 80).

WARNINGS & PRECAUTIONS

Hypersensitivity Reactions

Hypersensitivity reactions are possible with ADYNOVATE. Allergic-type
hypersensitivity reactions, including anaphylaxis, have been reported
with other recombinant antihemophilic factor VIII products, including
the parent molecule, ADVATE. Early signs of hypersensitivity reactions
that can progress to anaphylaxis may include angioedema, chest
tightness, dyspnea, wheezing, urticaria, and pruritus. Immediately
discontinue administration and initiate appropriate treatment if
hypersensitivity reactions occur.

Neutralizing Antibodies

Formation of neutralizing antibodies (inhibitors) to factor VIII can
occur following administration of ADYNOVATE. Monitor patients regularly
for the development of factor VIII inhibitors by appropriate clinical
observations and laboratory tests. Perform an assay that measures factor
VIII inhibitor concentration if the plasma factor VIII level fails to
increase as expected, or if bleeding is not controlled with expected
dose.

ADVERSE REACTIONS

Common adverse reactions (=1% of subjects) reported in the clinical
studies were headache and nausea.

ADVATE has a demonstrated efficacy and safety profile for the treatment
of hemophilia A. ADVATE is a full-length (derived from the complete
FVIII gene) recombinant FVIII product that is processed without any
blood-based additives. Because no blood-derived components are added at
any stage of the manufacturing process, the potential risk of
transmitting pathogens that may be carried in blood-based additives is
virtually eliminated. There have been no confirmed reports of
transmission of HIV, HBV or HCV with rFVIII treatments.

Neutralizing antibodies (inhibitors) have been reported following
administration of ADVATE predominantly in previously untreated patients
(PUPs) and previously minimally treated patients (MTPs). Monitor all
patients for the development of factor VIII inhibitors by appropriate
clinical observation and laboratory testing. If expected plasma factor
VIII activity levels are not attained, or if bleeding is not controlled
with an expected dose, perform an assay that measures factor VIII
inhibitor concentration.

ADVERSE REACTIONS

Serious adverse reactions seen with ADVATE are hypersensitivity
reactions, including anaphylaxis, and the development of high-titer
inhibitors necessitating alternative treatments to factor VIII.

Baxalta Incorporated (NYSE: BXLT) is a global biopharmaceutical leader
developing, manufacturing and commercializing therapies for orphan
diseases and underserved conditions in hematology, immunology and
oncology. Driven by passion to make a meaningful impact on patients’
lives, Baxalta’s broad and diverse pipeline includes biologics with
novel mechanisms and advanced technology platforms such as gene therapy.
Launched in 2015 following separation from Baxter International,
Baxalta’s heritage in biopharmaceuticals spans decades. Baxalta’s
therapies are available in more than 100 countries and it has advanced
biological manufacturing operations across 12 facilities, including
state-of-the-art recombinant production and plasma fractionation.
Headquartered in Northern Illinois, with its Global Innovation Center in
Cambridge, Mass., Baxalta employs 17,000 employees worldwide.

Forward-Looking Statements

This release includes forward-looking statements concerning
ADYNOVATE, including expectations with regard to regulatory filings and
potential impact on patients. Such statements are made of the date that
they were first issued and are based on current expectations, beliefs
and assumptions of management. Forward-looking statements are subject to
a number of risks and uncertainties, many of which involve factors or
circumstances that are beyond Baxalta's control and which could cause
actual results to differ materially from those in the forward-looking
statements, including the following: satisfaction of regulatory and
other requirements; actions of regulatory bodies and other governmental
authorities; changes in laws and regulations; product quality,
manufacturing or supply issues; patient safety issues; and other risks
identified in Baxalta's filings with the Securities and Exchange
Commission, all of which are available on Baxalta's website. Baxalta
expressly disclaims any intent or obligation to update these
forward-looking statements except as required by law.

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